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Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders

BACKGROUND: Major efforts have been made in the last decade to develop and improve therapies for proximal spinal muscular atrophy (SMA). The introduction of Nusinersen/Spinraza™ as an antisense oligonucleotide therapy, Onasemnogene abeparvovec/Zolgensma™ as an AAV9-based gene therapy and Risdiplam/E...

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Detalles Bibliográficos
Autores principales:	Jablonka, Sibylle, Hennlein, Luisa, Sendtner, Michael
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2022
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8725368/ https://www.ncbi.nlm.nih.gov/pubmed/34983696 http://dx.doi.org/10.1186/s42466-021-00162-9

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